Hemophilia Inhibitor PUP Study
- Conditions
- Bleeding disorderHemophilia A10064477
- Registration Number
- NL-OMON39820
- Lead Sponsor
- niversity of Texas Health Science Center
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 2
1. An informed consent, approved by the appropriate Institutional Review Board (IRB)/Independent Ethics Committee (IEC), has been administered, signed, and dated.
2. Subject has severe hemophilia A defined by a baseline FVIII:C <0.01 IU/ml. His FVIII activity will be confirmed at the central laboratory. If the confirmatory level is >=0.01 IU/ml the child must exit the study.
3. Subject weighs 3.5 kg or more at the time of his baseline study evaluation
1. Subject has had prior exposure to clotting factor concentrates or blood products, including packed red blood cells (RBC), platelets, plasma, or cryoprecipitate.
2. Subject has a clinically significant chronic disease other than hemophilia A.
3. Subject is currently participating in another investigational drug study.
Study & Design
- Study Type
- Observational invasive
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Primary Endpoints:<br /><br>An inhibitor is defined by a Nijmegen test > 0.6 Bethesda units (BU) on two<br /><br>consecutive tests conducted in the central laboratory.<br /><br>During the first 50 days of exposure to a single FVIII product:<br /><br>- Analyze and quantify subclasses of anti-FVIII antibodies<br /><br>- Characterize FVIII-specific T-cells and changes which occur<br /><br>- Quantify total FOXP3-positive regulatory T-cells (Treg)<br /><br>- Assess RNA expression, transcript profile, and exon usage in relevant pathways<br /><br>- Identify F8 gene mutation and other known genomic predictors of inhibitor<br /><br>development<br /><br>- Record infection(s), immunization(s), bleeding episodes, and factor usage</p><br>
- Secondary Outcome Measures
Name Time Method <p>N.a.</p><br>